Published in Mol Ther on July 24, 2007
Sustained alpha-sarcoglycan gene expression after gene transfer in limb-girdle muscular dystrophy, type 2D. Ann Neurol (2010) 2.02
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Evaluation of vascular delivery methodologies to enhance rAAV6-mediated gene transfer to canine striated musculature. Mol Ther (2009) 1.13
Changes in muscle T2 and tissue damage after downhill running in mdx mice. Muscle Nerve (2011) 1.13
Relative persistence of AAV serotype 1 vector genomes in dystrophic muscle. Genet Vaccines Ther (2008) 0.97
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Treatment of leber congenital amaurosis due to RPE65 mutations by ocular subretinal injection of adeno-associated virus gene vector: short-term results of a phase I trial. Hum Gene Ther (2008) 8.83
Human mesenchymal stem cells differentiate to a cardiomyocyte phenotype in the adult murine heart. Circulation (2002) 7.97
Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics. Proc Natl Acad Sci U S A (2008) 7.16
Defective membrane repair in dysferlin-deficient muscular dystrophy. Nature (2003) 6.50
Targeting misperceptions of descriptive drinking norms: efficacy of a computer-delivered personalized normative feedback intervention. J Consult Clin Psychol (2004) 6.11
Production and purification of serotype 1, 2, and 5 recombinant adeno-associated viral vectors. Methods (2002) 5.30
Post-translational disruption of dystroglycan-ligand interactions in congenital muscular dystrophies. Nature (2002) 5.21
Are social norms the best predictor of outcomes among heavy-drinking college students? J Stud Alcohol Drugs (2007) 4.75
Deletion of brain dystroglycan recapitulates aspects of congenital muscular dystrophy. Nature (2002) 3.96
Dystroglycan: from biosynthesis to pathogenesis of human disease. J Cell Sci (2006) 3.92
Next generation of adeno-associated virus 2 vectors: point mutations in tyrosines lead to high-efficiency transduction at lower doses. Proc Natl Acad Sci U S A (2008) 3.84
Adult hematopoietic stem cells provide functional hemangioblast activity during retinal neovascularization. Nat Med (2002) 3.81
Gene therapy for leber congenital amaurosis caused by RPE65 mutations: safety and efficacy in 15 children and adults followed up to 3 years. Arch Ophthalmol (2011) 3.79
Limb-girdle muscular dystrophy type 2D gene therapy restores alpha-sarcoglycan and associated proteins. Ann Neurol (2009) 3.69
Recombinant adeno-associated virus serotype 9 leads to preferential cardiac transduction in vivo. Circ Res (2006) 3.63
Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy. Proc Natl Acad Sci U S A (2009) 3.58
Cell therapy of alpha-sarcoglycan null dystrophic mice through intra-arterial delivery of mesoangioblasts. Science (2003) 3.21
Muscular dystrophies involving the dystrophin-glycoprotein complex: an overview of current mouse models. Curr Opin Genet Dev (2002) 3.15
Normative misperceptions and temporal precedence of perceived norms and drinking. J Stud Alcohol (2006) 3.12
Auxiliary subunits: essential components of the voltage-gated calcium channel complex. Curr Opin Neurobiol (2003) 3.10
Human RPE65 gene therapy for Leber congenital amaurosis: persistence of early visual improvements and safety at 1 year. Hum Gene Ther (2009) 3.07
Structural analysis of the voltage-dependent calcium channel beta subunit functional core and its complex with the alpha 1 interaction domain. Neuron (2004) 3.05
Celebration intoxication: an evaluation of 21st birthday alcohol consumption. J Am Coll Health (2005) 3.04
Pompe disease diagnosis and management guideline. Genet Med (2006) 3.03
LARGE can functionally bypass alpha-dystroglycan glycosylation defects in distinct congenital muscular dystrophies. Nat Med (2004) 3.00
O-mannosyl phosphorylation of alpha-dystroglycan is required for laminin binding. Science (2010) 2.98
Being controlled by normative influences: self-determination as a moderator of a normative feedback alcohol intervention. Health Psychol (2006) 2.96
Sarcolemma-localized nNOS is required to maintain activity after mild exercise. Nature (2008) 2.87
PGC-1alpha regulates the neuromuscular junction program and ameliorates Duchenne muscular dystrophy. Genes Dev (2007) 2.79
Vision 1 year after gene therapy for Leber's congenital amaurosis. N Engl J Med (2009) 2.57
Dystrophin-glycoprotein complex: post-translational processing and dystroglycan function. J Biol Chem (2003) 2.56
Internet-based personalized feedback to reduce 21st-birthday drinking: a randomized controlled trial of an event-specific prevention intervention. J Consult Clin Psychol (2009) 2.55
Dysferlin and the plasma membrane repair in muscular dystrophy. Trends Cell Biol (2004) 2.41
Are voltage-dependent ion channels involved in the endothelial cell control of vasomotor tone? Am J Physiol Heart Circ Physiol (2007) 2.41
Efficacy of web-based personalized normative feedback: a two-year randomized controlled trial. J Consult Clin Psychol (2010) 2.35
New World arenavirus clade C, but not clade A and B viruses, utilizes alpha-dystroglycan as its major receptor. J Virol (2002) 2.35
Molecular recognition by LARGE is essential for expression of functional dystroglycan. Cell (2004) 2.33
Dystroglycan function requires xylosyl- and glucuronyltransferase activities of LARGE. Science (2012) 2.22
Posttranslational modification of alpha-dystroglycan, the cellular receptor for arenaviruses, by the glycosyltransferase LARGE is critical for virus binding. J Virol (2005) 2.17
Abnormal coronary function in mice deficient in alpha1H T-type Ca2+ channels. Science (2003) 2.05
Sustained alpha-sarcoglycan gene expression after gene transfer in limb-girdle muscular dystrophy, type 2D. Ann Neurol (2010) 2.02
The unfolded protein response mediates adaptation to exercise in skeletal muscle through a PGC-1α/ATF6α complex. Cell Metab (2011) 2.02
Disruption of DAG1 in differentiated skeletal muscle reveals a role for dystroglycan in muscle regeneration. Cell (2002) 2.01
The relative impact of injunctive norms on college student drinking: the role of reference group. Psychol Addict Behav (2008) 1.96
Tyrosine-phosphorylation of AAV2 vectors and its consequences on viral intracellular trafficking and transgene expression. Virology (2008) 1.96
RIM1 confers sustained activity and neurotransmitter vesicle anchoring to presynaptic Ca2+ channels. Nat Neurosci (2007) 1.95
Event- and context-specific normative misperceptions and high-risk drinking: 21st birthday celebrations and football tailgating. J Stud Alcohol (2006) 1.94
ISPD loss-of-function mutations disrupt dystroglycan O-mannosylation and cause Walker-Warburg syndrome. Nat Genet (2012) 1.93
Event-specific drinking among college students. Psychol Addict Behav (2011) 1.91
Dysferlin-mediated membrane repair protects the heart from stress-induced left ventricular injury. J Clin Invest (2007) 1.88
Phase I trial of intramuscular injection of a recombinant adeno-associated virus serotype 2 alphal-antitrypsin (AAT) vector in AAT-deficient adults. Hum Gene Ther (2006) 1.83
Dysferlin and muscle membrane repair. Curr Opin Cell Biol (2007) 1.82
Acid alpha-glucosidase deficiency (glycogenosis type II, Pompe disease). Curr Mol Med (2002) 1.82
Unique role of dystroglycan in peripheral nerve myelination, nodal structure, and sodium channel stabilization. Neuron (2003) 1.80
Sarcolemmal-restricted localization of functional ClC-1 channels in mouse skeletal muscle. J Gen Physiol (2010) 1.73
Evaluating level of specificity of normative referents in relation to personal drinking behavior. J Stud Alcohol Drugs Suppl (2009) 1.73
Group identification as a moderator of the relationship between perceived social norms and alcohol consumption. Psychol Addict Behav (2010) 1.68
Preliminary examination of spring break alcohol use and related consequences. Psychol Addict Behav (2009) 1.68
A dystroglycan mutation associated with limb-girdle muscular dystrophy. N Engl J Med (2011) 1.67
Neural deficits contribute to respiratory insufficiency in Pompe disease. Proc Natl Acad Sci U S A (2009) 1.67
Cardiac and clinical phenotype in Barth syndrome. Pediatrics (2006) 1.61
Limb-girdle muscular dystrophy in the United States. J Neuropathol Exp Neurol (2006) 1.59
SGK196 is a glycosylation-specific O-mannose kinase required for dystroglycan function. Science (2013) 1.57
Phase I trial of intramuscular injection of a recombinant adeno-associated virus alpha 1-antitrypsin (rAAV2-CB-hAAT) gene vector to AAT-deficient adults. Hum Gene Ther (2004) 1.54
Transcriptional upregulation of Cav3.2 mediates epileptogenesis in the pilocarpine model of epilepsy. J Neurosci (2008) 1.54
Gene therapy for rare diseases: summary of a National Institutes of Health workshop, September 13, 2012. Hum Gene Ther (2013) 1.54
LateTIME: a phase-II, randomized, double-blinded, placebo-controlled, pilot trial evaluating the safety and effect of administration of bone marrow mononuclear cells 2 to 3 weeks after acute myocardial infarction. Tex Heart Inst J (2010) 1.52
Characterization of a recombinant adeno-associated virus type 2 Reference Standard Material. Hum Gene Ther (2010) 1.51
Mutations in GDP-mannose pyrophosphorylase B cause congenital and limb-girdle muscular dystrophies associated with hypoglycosylation of α-dystroglycan. Am J Hum Genet (2013) 1.50
Dystroglycan loss disrupts polarity and beta-casein induction in mammary epithelial cells by perturbing laminin anchoring. J Cell Sci (2006) 1.49
A randomized controlled trial of event-specific prevention strategies for reducing problematic drinking associated with 21st birthday celebrations. J Consult Clin Psychol (2012) 1.48
Point mutation in the glycoprotein of lymphocytic choriomeningitis virus is necessary for receptor binding, dendritic cell infection, and long-term persistence. Proc Natl Acad Sci U S A (2011) 1.47
C-terminal titin deletions cause a novel early-onset myopathy with fatal cardiomyopathy. Ann Neurol (2007) 1.47
Basal lamina strengthens cell membrane integrity via the laminin G domain-binding motif of alpha-dystroglycan. Proc Natl Acad Sci U S A (2009) 1.47
Mutations in B3GALNT2 cause congenital muscular dystrophy and hypoglycosylation of α-dystroglycan. Am J Hum Genet (2013) 1.47
Loss of alpha-dystroglycan laminin binding in epithelium-derived cancers is caused by silencing of LARGE. J Biol Chem (2009) 1.46
Exercise-induced left ventricular systolic dysfunction in women heterozygous for dystrophinopathy. J Am Soc Echocardiogr (2010) 1.43
Structural determinants of tissue tropism and in vivo pathogenicity for the parvovirus minute virus of mice. J Virol (2005) 1.41
Pompe disease gene therapy. Hum Mol Genet (2011) 1.39
Social motives and the interaction between descriptive and injunctive norms in college student drinking. J Stud Alcohol Drugs (2007) 1.37
Cardiac overexpression of angiotensin converting enzyme 2 protects the heart from ischemia-induced pathophysiology. Hypertension (2008) 1.37
Transcriptional pathways associated with skeletal muscle disuse atrophy in humans. Physiol Genomics (2007) 1.35
The Ca(v)3.2 T-type Ca(2+) channel is required for pressure overload-induced cardiac hypertrophy in mice. Circ Res (2009) 1.34
Fukutin gene mutations cause dilated cardiomyopathy with minimal muscle weakness. Ann Neurol (2006) 1.34
The voltage-dependent calcium channel beta subunit contains two stable interacting domains. J Biol Chem (2003) 1.33
Dystroglycan is selectively associated with inhibitory GABAergic synapses but is dispensable for their differentiation. J Neurosci (2002) 1.33
Proteolytic enzymes and altered glycosylation modulate dystroglycan function in carcinoma cells. Cancer Res (2004) 1.33
Glycomic analyses of mouse models of congenital muscular dystrophy. J Biol Chem (2011) 1.32
Like-acetylglucosaminyltransferase (LARGE)-dependent modification of dystroglycan at Thr-317/319 is required for laminin binding and arenavirus infection. Proc Natl Acad Sci U S A (2011) 1.32
Physiological correction of Pompe disease by systemic delivery of adeno-associated virus serotype 1 vectors. Mol Ther (2007) 1.31
Both laminin and Schwann cell dystroglycan are necessary for proper clustering of sodium channels at nodes of Ranvier. J Neurosci (2005) 1.30
Successful production of pseudotyped rAAV vectors using a modified baculovirus expression system. Mol Ther (2005) 1.30
Improved method of recombinant AAV2 delivery for systemic targeted gene therapy. Mol Ther (2002) 1.29